EntreMed, Inc. was granted orphan drug designation by the FDA for its lead oncology drug candidate, ENMD-2076, for the treatment of acute myeloid leukemia (AML). Initial results from their Phase 1 study demonstrated that ENMD-2076, administered orally as a single agent, was well tolerated in this patient population. The data for 15 evaluable AML patients showed that one patient achieved a CRi (complete remission with incomplete hematological recovery) and two patients achieved a morphologic leukemia-free state (MLFS). Three additional patients participating in the study experienced 11%, 14%, and 65% reductions in marrow blast count.

ENMD-2076 is an orally-active, Aurora A/angiogenic kinase inhibitor with a unique kinase selectivity profile and multiple mechanisms of action.